Corporate Banner
Satellite Banner
Biologics & Bioprocessing
Scientific Community
Become a Member | Sign in
Home>News>This Article

Agilis and Intrexon Collaborate on Rare Diseases

Published: Friday, January 03, 2014
Last Updated: Friday, January 03, 2014
Bookmark and Share
Companies announce exclusive channel collaboration (ECC) to develop DNA-based therapeutics for Friedreich's ataxia (FRDA), a rare genetic neurodegenerative disease.

Agilis and Intrexon expect DNA-based therapeutics to provide the ability to target underlying disease mechanisms with precision using tightly-controlled gene therapies for patients with rare inherited diseases such as FRDA.  Current FRDA therapies are primarily focused on supportive care and symptom relief.  There are no FDA-approved treatment options to address the cause of FRDA.

The technical core of Agilis' novel DNA-based therapeutics will utilize Intrexon's UltraVector® platform and RheoSwitch Therapeutic System® (RTS®) to develop gene therapies and genetically-modified cell therapies for treating FRDA. The ECC's planned approach to target FRDA will employ RTS®, a clinically validated inducible gene switch technology that regulates the expression of therapeutic proteins or bioactive RNA in a dose-dependent fashion.

FRDA is an inherited disease caused by a gene mutation that reduces the expression of frataxin, a protein localized in the "power center" of cells known as the mitochondria, and results in a physically debilitating, life-shortening condition.  Progression of the disease causes nervous system damage, problems with movement and early death most often caused by cardiac malfunction.   FRDA is the most common hereditary ataxia with an estimated 5,000 to 10,000 patients in the United States.   

The goal of the ECC is to develop DNA-based therapeutics to repair or replace the "broken" gene in FRDA and enable increased production of the frataxin protein to alleviate the downstream effects of frataxin deficiency. The combination of gene correction with additional therapeutic modulators in a multigenic approach has the potential to create a potent treatment.  This treatment is expected to further improve cardiovascular and neurological function by addressing the underlying causes of FRDA and associated symptoms.  Agilis also has an option to advance a second undisclosed rare disease indication.

"We are thrilled to be working with Intrexon with the hope of providing children and adults affected with rare genetic disorders with promising new treatments," said George S. Zorich, Chief Executive Officer of Agilis Biotherapeutics.  "We believe Agilis is on the forefront of one of the most promising treatment breakthroughs for Friedreich's ataxia. I am personally excited to collaborate with the Intrexon team and look forward to developing new transformative therapies together."

Samuel Broder, M.D., Senior Vice President of Intrexon's Health Sector and former Director of the National Cancer Institute, said, "FRDA causes heart failure and progressive neurological deterioration, which in turn cause suffering and premature deaths.  The goal of this collaboration is to harnessIntrexon's proprietary technologies in synthetic DNA, as well as our expertise in molecular, protein, and cellular engineering, to benefit patients with this very serious disorder." 

Intrexon Founder and Chief Science Officer Thomas Reed, Ph.D., remarked that unlike some biotechnology companies which are limited to single classes of therapeutic molecules or gene delivery systems, Intrexon is equipped to pursue several different therapeutic approaches for treating the complexities associated with FRDA. 

"As an inventor and integrator of technology platforms, we will make use of our UltraVector® multi-gene engineering, RTS® gene switch, advanced Protein Engineering, and other platforms to develop therapeutic candidates designed to treat both the neurological and the cardiovascular pathologies of FRDA.  We are confident that our ability to pursue multiple approaches for treating this complex disease will significantly increase our probability of success.

Further Information

Join For Free

Access to this exclusive content is for Technology Networks Premium members only.

Join Technology Networks Premium for free access to:

  • Exclusive articles
  • Presentations from international conferences
  • Over 3,500+ scientific posters on ePosters
  • More than 5,100+ scientific videos on LabTube
  • 35 community eNewsletters

Sign In

Forgotten your details? Click Here
If you are not a member you can join here

*Please note: By logging into you agree to accept the use of cookies. To find out more about the cookies we use and how to delete them, see our privacy policy.

Scientific News
Mass Spec Technology Drives Innovation Across the Biopharma Workflow
With greater resolving power, analytical speed, and accuracy, new mass spectrometry technology and techniques are infiltrating the biopharmaceuticals workflow.
Overlooked Molecules Could Revolutionise our Understanding of the Immune System
Researchers have discovered that around one third of all the epitopes displayed for scanning by the immune system are a type known as ‘spliced’ epitopes.
Signaling Pathway Could Be Key to Improved Osteoporosis Treatment
Inhibition of SIK2 enzyme both stimulates bone formation and reduces bone breakdown in animal model.
Less Frequent Cervical Cancer Screening
HPV-vaccinated women may only need one screening every 5 to 10 years with screening starting later in life.
The Power Of Orthogonality In Assessing The Stability Of Biopharmaceuticals
By utilizing orthogonal techniques, researchers can maximize the secure application of all analytical results generated.
Dysfunction in Neuronal Transport Mechanism Linked to Alzheimer’s
Findings confirm mutation-caused problem but also reveal a new therapeutic target.
New Antibody Therapy Permanently Blocks SIV Infection
An international research team has developed an effective treatment strategy against the HIV-like Simian Immunodeficiency Virus (SIV) in rhesus macaques.
3D-Printing in Science: Conference Co-Staged with LABVOLUTION
LABVOLUTION 2017 will have an added highlight of a simultaneous conference, "3D-Printing in Science".
DNA Vaccines Protect Monkeys Against Zika Virus
Two experimental Zika virus DNA vaccines developed by NIH scientists protected monkeys against Zika infection.
Rare Flu-Thwarting Mutation Discovered
Study finds protein mutation, that is encoded by influenza, causes the virus to lose any defence against the immune system.
Skyscraper Banner

SELECTBIO Market Reports
Go to LabTube
Go to eposters
Access to the latest scientific news
Exclusive articles
Upload and share your posters on ePosters
Latest presentations and webinars
View a library of 1,800+ scientific and medical posters
3,500+ scientific and medical posters
A library of 2,500+ scientific videos on LabTube
5,100+ scientific videos